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S. HRG.

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STEM CELLS RESEARCH, 2005

HEARING
BEFORE A

SUBCOMMITTEE OF THE

COMMITTEE ON APPROPRIATIONS
UNITED STATES SENATE
ONE HUNDRED NINTH CONGRESS
FIRST SESSION

SPECIAL HEARING
OCTOBER 19, 2005WASHINGTON, DC

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COMMITTEE ON APPROPRIATIONS
THAD COCHRAN, Mississippi, Chairman
TED STEVENS, Alaska
ROBERT C. BYRD, West Virginia
ARLEN SPECTER, Pennsylvania
DANIEL K. INOUYE, Hawaii
PETE V. DOMENICI, New Mexico
PATRICK J. LEAHY, Vermont
CHRISTOPHER S. BOND, Missouri
TOM HARKIN, Iowa
MITCH MCCONNELL, Kentucky
BARBARA A. MIKULSKI, Maryland
CONRAD BURNS, Montana
HARRY REID, Nevada
RICHARD C. SHELBY, Alabama
HERB KOHL, Wisconsin
JUDD GREGG, New Hampshire
PATTY MURRAY, Washington
ROBERT F. BENNETT, Utah
BYRON L. DORGAN, North Dakota
LARRY CRAIG, Idaho
DIANNE FEINSTEIN, California
KAY BAILEY HUTCHISON, Texas
RICHARD J. DURBIN, Illinois
MIKE DEWINE, Ohio
TIM JOHNSON, South Dakota
SAM BROWNBACK, Kansas
MARY L. LANDRIEU, Louisiana
WAYNE ALLARD, Colorado
J. KEITH KENNEDY, Staff Director
TERRENCE E. SAUVAIN, Minority Staff Director

SUBCOMMITTEE

ON

DEPARTMENTS OF LABOR, HEALTH AND HUMAN SERVICES,


EDUCATION, AND RELATED AGENCIES

AND

ARLEN SPECTER, Pennsylvania, Chairman


THAD COCHRAN, Mississippi
TOM HARKIN, Iowa
JUDD GREGG, New Hampshire
DANIEL K. INOUYE, Hawaii
LARRY CRAIG, Idaho
HARRY REID, Nevada
KAY BAILEY HUTCHISON, Texas
HERB KOHL, Wisconsin
TED STEVENS, Alaska
PATTY MURRAY, Washington
MIKE DEWINE, Ohio
MARY L. LANDRIEU, Louisiana
RICHARD C. SHELBY, Alabama
RICHARD J. DURBIN, Illinois
ROBERT C. BYRD, West Virginia (Ex officio)
Professional Staff
BETTILOU TAYLOR
JIM SOURWINE
MARK LAISCH
SUDIP SHRIKANT PARIKH
CANDICE ROGERS
LISA BERNHARDT
RACHEL JONES
ELLEN MURRAY (Minority)
ERIK FATEMI (Minority)
ADRIENNE HALLETT (Minority)

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CONTENTS
Page

Opening statement of Senator Arlen Specter ........................................................


Statement of Senator Mary L. Landrieu ...............................................................
Prepared statement ..........................................................................................
Statement of Senator Tom Harkin .........................................................................
Statement of Senator Thad Cochran ......................................................................
Statement of Anthony Herrera, author and cancer survivor ...............................
Prepared statement ..........................................................................................
Statement of Judith Gasson, Ph.D., director, Jonsson Comprehensive Cancer
Center ....................................................................................................................
Prepared statement ..........................................................................................
Statement of Rudolf Jaenisch, M.D., professor of biology, Massachusetts Institute of Technology ................................................................................................
Prepared statement ..........................................................................................
Statement of Steven Teitelbaum, M.D., Wilma and Roswell Messing, professor
of Pathology and Immunology, Washington University School of Medicine ...
Prepared statement ..........................................................................................
Statement of John Wagner, M.D., scientific director of clinical research, Blood
and Marrow Transplant Program and Stem Cell Institute ..............................
Prepared statement ..........................................................................................

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STEM CELLS RESEARCH, 2005


WEDNESDAY, OCTOBER 19, 2005

U.S. SENATE,
SUBCOMMITTEE ON LABOR, HEALTH AND HUMAN
SERVICES, EDUCATION, AND RELATED AGENCIES,
COMMITTEE ON APPROPRIATIONS,
Washington, DC.
The subcommittee met at 9:31 a.m., in room SD138, Dirksen
Senate Office Building, Hon. Arlen Specter (chairman) presiding.
Present: Senators Specter, Cochran, Harkin, and Landrieu.
OPENING STATEMENT OF SENATOR ARLEN SPECTER

Senator SPECTER. Good morning.


Today our focus is on stem cells. Our hearing coincides with the
announcement by the South Korean Nuclear Transplantation Consortium that they are starting an operation today on a world stem
cell foundation which will be based in the Seoul National University in South Korea with satellite offices in San Francisco and England. I applaud what they are doing, but I regret that the United
States is falling farther behind in world leadership on scientific research generally and specifically on stem cell research.
Now, this is the 17th hearing which this subcommittee has held
on this important subject since we first learned about stem cells in
November 1998. It is well known that the stem cells have the possibility for curing or alleviating the problems of so many maladies.
The House of Representatives has passed legislation which would
remove the restriction by the Federal Government on funding stem
cell research, and Senator Harkin and I have introduced identical
legislation in the Senate, S. 471. The House bill received support
from some 50 Republicans crossing party lines, and it is my view
that with sufficient focus and sufficient attention, there could be
amassed enough votes to override a presidential veto. I say that regretfully and reluctantly, but this is a matter of utmost importance
and has a direct impact to some 110 million Americans.
President Nixon declared war on cancer in 1970 and had the
United States devoted the resources to that war which we devote
to other wars, I think the war on cancer could have been won. I
say that with special concern because I have had my own problems
with Hodgkins lymphoma cancer this year, and cancer continues
to claim deaths in the hundreds of thousands.
This happens to be an especially busy day. Most days are busy
on Capitol Hill, but we are in the midst of proceedings on the nomination of Ms. Miers for the Supreme Court, and I am going to have
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to excuse myself at about 10:00, but I will have an opportunity before that occurs to hear all of the witnesses.
Now I would like to yield to my distinguished ranking member,
Senator Harkin.
Senator HARKIN. Thank you very much, Mr. Chairman. Senator
Landrieu I know has to leave right away. If I could just yield a couple minutes for her of my time, I would appreciate it.
Senator SPECTER. Well, I was about to yield a couple minutes to
Senator Cochran, but since you spoke first, Senator Landrieu.
STATEMENT OF SENATOR MARY L. LANDRIEU

Senator LANDRIEU. Thank you, Mr. Chairman. I thank Senator


Harkin just for one moment because I have got to leave for another
meeting. It is a very busy day, as the chairman said.
Let me thank the chairman and Senator Harkin for their pursuit
of a solution to this dilemma and to this great challenge. I have
a slightly different view that I will submit for the record in written
testimony.
For this morning, I will just say that as we pursue cures for the
many diseases that challenge us and while I understand that embryonic stem cells hold promise for curing diseases, as the chairman and many others have pointed out, I think that we have to
be very mindful of what many of our ethical leaders have said and
the Catholic bishops in particular that it is important in the pursuit of progress to not undermine human dignity. And there is a
line that can be drawn between progress and human dignity, and
creating embryonic stem cells for the purpose of creating human
beings for the purposes of destroying them for science crosses that
line in my opinion.
PREPARED STATEMENT

I will submit more to the record, but I understand that this will
be a continued debate, and I thank the Senator for allowing me to
express my views.
Senator SPECTER. Thank you, Senator Landrieu.
[The statement follows:]
PREPARED STATEMENT

OF

SENATOR MARY L. LANDRIEU

Thank you, Mr. Chairman. I have had the privilege of serving as a member of
this subcommittee for four years now. I think it is important to note that one of
the very first hearings I attended was on this very issue. A lot has changed since
thenboth in the ethical debate and in the science. But what have not changed are
the moral parameters that must guide us in these decisions. As Richard Doerflinger
of the Catholic Conference of Bishops put itWe must be careful not to undermine
human dignity in the pursuit of human progress.
Since that hearing four years ago, in August of 2001, the President issued an executive order, allowing for federal funding for stem cell research on the then existing stem cell lines. In November of that same year, he appointed a council to monitor stem cell research, to recommend appropriate guidelines and regulations, and
to consider all of the medical and ethical ramifications of biomedical innovation. To
date, this council has issued six hundred plus page reports on the bioethics issues
involved in stem cell research. Meanwhile, the scientific community has moved forward in its advancements in knowledge and discovery. And everyday we, as members of Congress are faced with the questions of how far we should go in the name
of science.
There is no doubt that embryonic stem cell research holds the promise of curing
diseases such as Parkinsons, diabetes, Alzheimers and cancer. Even President
Bush stressed the importance of federally funded research in approving the original

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stem cell lines in 2001he explicitly stated that federal dollars help attract the best
and brightest scientists and help ensure that new discoveries are widely shared at
the largest number of research facilities. Federal funding not only allows us to encourage and financially support this research, it allows us to use the power of the
purse to be sure it is done in the most safe and ethical way possible. Mr. Chairman,
I want to state clearly for the record, I support federal funding for embryonic stem
cell research provided that the embryos used in these studies are those that are in
excess from the fertility process and are knowingly donated for this purpose.
I have met with many constituents suffering from life altering and fatal diseases
and they have told me the impact that this research may have on their lives. One
such constituent who I will never forget is a nine year old girl, Sarah, who suffers
from juvenile diabetes. Sarah told me of her daily routine of shots and blood tests.
Her parents told me of some of the effects of diabetes such as vision loss, kidney
failure, blindness, nerve damage, amputations, heart attack, and stroke. They
begged me, on her behalf, not to block this important research that could mean a
normal childhood for Sarah. Sarah is not alone in this hope, 35 children a day are
diagnosed with Type One Diabetes.
There are currently 400,000 frozen embryos at IVF clinics around the country
88.2 percent of which are used for implantation in the mothers womb2.8 percent
are given up for adoptionthe wonderful snowflake babies we all hear so much
about. This translates to a total of 11,000 embryos that are not going to be implanted and are voluntarily donated. It is important to note, if these embryos were
not donated then they would be destroyednot for sciencebut thrown away with
the rest of the medical waste for the day. We cannot allow these valuable embryos
to be discarded when even according to the Presidents Council on Bioethics, stem
cells and their derivatives may prove a valuable source of transplantable cells and
tissues for repair and regeneration. If these healing powers could be harnessed, the
medical benefits for humankind would be immense, perhaps ushering in an era of
truly regenerative medicine.
Please do no let my views expressed today confuse your understanding of my support of legislation banning human cloning. Embryonic stem cell research using excess embryos from IVF treatments and creating cloned embryos for scientific purposes should not be confused. I believe that creating a human embryo for the sole
purpose of its destruction through experimentation is wrong, unethical and should
be illegal. Since I mentioned the hearing on stem cell research conducted by this
committee four years ago at the beginning of my remarks, I think it is important
to note that many members of this subcommittee also expressed concerns about the
creation of human embryos for research.
The human body is not a product to be mass-produced and stripped for parts,
most especially in the earliest stages of its developmentwomens eggs and wombs
should never be commodities sold to the highest bidders. But this is a very real risk
of so called therapeutic cloning. Experts estimate that over 800 million eggs would
be needed to support one-sixteenth of the possible human cloning experiments. We
are already getting reports that clinical researchers in Seoul, Korea, in England,
and in San Francisco will be working with the South Korean veterinarian and stemcell biologist whose laboratory leads the world in the use of the somatic-cell nuclear
transfer technique, to recruit women to donate eggs and patients to donate somatic
cells.
Whats more, regardless of what proponents of this research will tell you, there
is only one kind of cloning. The only difference between what has come to be called
reproductive cloning and therapeutic cloning is what is done with the clones once
they are created. Legislation that purports to ban the birth of a cloned human being
does not ban its creation, only its implantation into a human uterus. Once we support and encourage the creation of millions of cloned human beings, do we really
believe we would have the power to successfully monitor and ban their implantation? The only effective way to ban human cloning is to stop the process before it
starts.
Finally, Mr. Chairman, it is because I believe that there is immense potential in
embryonic and adult stem cell research that I oppose federal support for human
cloning. I believe that banning, even if only temporarily, this one procedure helps
to focus science and funding for research to equally promising but less problematic
areas such as embryonic and adult stem cell research.
I look forward to hearing from the witnesses today and thank you, Chairman
Specter for holding this important hearing.

Senator SPECTER. Senator Harkin.

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STATEMENT OF SENATOR TOM HARKIN

Senator HARKIN. Thank you, Mr. Chairman. Again, let me just


compliment you on your great leadership on a lot of things, but especially on this issue since 1998, as you mentioned, and for calling
this hearing.
We have had a pretty busy year with hurricanes on the Gulf
Coast, of course, and as the chairman knows better than anyone
else, the Senate having to have hearings on two Supreme Court
nominees, which the chairman chairs that committee. And yet, the
need to continue our push for stem cell research is as critical as
ever.
I was privileged this summer to meet with some of the South Koreans, and you mentioned the article that was in the paper this
morning, Mr. Chairman, that they are moving ahead on this, the
whole area of somatic cell nuclear transfer, and the kind of promise
that holds.
I am just hopeful that we can move ahead on this. People are
suffering and dying. They need hope. We know this holds great
promise. We all know that medical research is not just done by one
person, not done by two. The best research is when a lot of people
are involved in it and it is spread around. That is what we need
to do. We need to get more involved in this type of research.
Yet, we have manacles put on our researchers today and we need
to remove those. That is what the bill that Senator Specter and I
have supported, the one that passed the House, does. That is why
we hope today we can move ahead with a look at somatic cell nuclear transfer and what that means for the future of stem cell research.
So, again, Mr. Chairman, I thank you for holding this hearing
and, again, I thank you for your great leadership on this issue.
Senator SPECTER. Senator Cochran, would you care to make an
opening statement?
STATEMENT OF SENATOR THAD COCHRAN

Senator COCHRAN. Mr. Chairman, thank you very much. I congratulate you on your continued strong leadership in helping to explore the possibilities that medical research holds for curing and
preventing illness and disease. You have done more than anybody
I can remember since I have been in the Senate to not only focus
attention on these opportunities that we have to legislate and support research and to authorize programs to achieve these goals. So
I congratulate you and thank you again for this hearing.
I am really here to introduce Anthony Herrera who is a friend
of mine since 1961, who is a member of this panel. So I will await
your advice as to when that would be appropriate.
Senator SPECTER. Well, thank you very much, Senator Cochran.
We appreciate your continuing support for this subcommittee and
your membership on the subcommittee, notwithstanding your very
onerous duties as chairman of the full Appropriations Committee.

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STATEMENT OF ANTHONY HERRERA, AUTHOR AND CANCER SURVIVOR

Senator SPECTER. We now move to our panel of witnesses and


our lead witness is Mr. Anthony Herrera. I yield to you again, Mr.
Chairman, for the formal introduction.
Senator COCHRAN. Thank you very much. It is a great pleasure
for me to introduce to the committee Anthony Herrera, whom I
have known since 1961. We met when I was entering my first year
of law school and he was beginning his first year of undergraduate
school at the University of Mississippi, and I happened to be in the
same residence hall and became the dormitory manager, as we
called it, back in those days. He was young, but energetic, full of
an interest in all of the things that were going on at that campus.
He became an excellent student and then went on to a successful
career in the performing arts as an actor, writer, director. He has
been on As the World Turns off and on for a long, long time. James
Stenbeck is his stage name on that show. And James Stenbeck has
been a survivor too of sorts. He would disappear. People would
think he was a goner and then he would reappear sometime later
full of life and enthusiasm. That is the story of Anthony Herrera
as well.
He has battled cancer and has survived. He has written a book
about it, The Cancer War, which I recommend. I know the chairman has read it. I have read it. It is very instructive into the challenges that confront someone who is a victim of lymphoma or other
forms of cancer. He had a rare kind of lymphoma. And transplants
of bone marrow, stem cells, all of these things have been involved
in his life. He has lived through it all and can help us understand
the challenges that victims face and the possible successes there
are in our effort to deal more successfully with some of these forms
of cancer.
So it is a great pleasure for me to welcome him and thank him
for being here to help us understand the challenges.
Senator SPECTER. Thank you very much, Mr. Chairman.
Mr. Herrera, we do very much appreciate your being here today,
especially since you came from Buenos Aires to participate in this
hearing. I compliment you on the book which you have written, and
I pay particular note to your references to stem cells as they relate
to your situation.
We now begin the customary 5-minute rounds for the witnesses
and we start with Mr. Anthony Herrera.
Mr. HERRERA. Thank you.
In January 1997 at New York Hospital, I was diagnosed with
mantle cell lymphoma and was told this disease will kill you. There
is nothing we can do. You are going to die.
Then without anesthesia, this oncologist drilled through my skin,
through my periosteum and into the bone, and extracted marrow.
The pain was incredible.
That night I debated whether to put my .38 Smith & Wesson to
my temple and pull the trigger or saddle up. I pondered each option. Then I pictured myself on a horse riding into a dark canyon.
I found a poem by Tennessee Williams from the Night of the Iguana. I read it every day.

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I then went to Sloan Kettering where I was told we are going to
work hard and hope for the best. They had a new protocol for mantle cell developed with a hospital in Paris. I was the fifth patient
in the United States to undergo this regimen, massive amounts of
chemotherapy and total body irradiation to kill lymphoma cells and
take my immune system to zero.
On August 1, 1997, I received an autologous stem cell transplant,
autologous meaning the stem cells were taken from my body. My
mouth was full of sores. My skin was gray. I had no hair, no fingernails, no toenails, but I was found to be in remission. I lived under
the belief that if the disease came back that I would die.
In November 1998, I relapsed but during these 18 months, a new
approach to the stem cell transplant for mantle cell had been developed at M.D. Anderson Cancer Center in Houston, Texas where I
was admitted March 30, 1999.
In April, I underwent an allogeneic stem cell transplant using a
non-myeloablative regimen, allogeneic meaning the cells came from
a donor, non-myeloablative meaning they did not burn my immune
system to zero with chemo, hence less toxicity.
Six weeks later, the lymphoma was still active. We tried a donor
lymphocyte infusion, adding more of my brothers cells. I then suffered from CMV. I bled internally and lost 30 pounds in 3 weeks,
followed by a mild stroke and a seizure, but I was in remission.
One year later, August 15, 2000, the CT-scan showed that I had
relapsed. The disease was back. I was told without treatment you
will die in less than 12 months and that another donor lymphocyte
infusion could kill you. There was a small amount of disease, so I
had time to think.
Six weeks later, I saddled up and requested a CT-scan. At this
juncture, medical history was made. This scan showed less disease
than 6 weeks before, which meant that my new immune system
had started fighting the lymphoma without chemotherapy, without
drugs, without radiation. My new immune system was taking out
the cancer, my new immune system and my bone marrow created
by donor stem cells.
Throughout this journey, I listened to Willie Nelson, Louis Armstrong, and Agustin Lara of Mexico. I recited Tennessee Williams
every day. I quoted from John Fords The Searchers. When asked
if he wanted to quit, John Wayne retorted, that will be the day.
I found dedicated and inspired doctors and nurses, such as Sergio
Giralt and Joyce Newman, doctors and nurses with guts and vision.
In 1950, William Faulkner won the Nobel Peace Prize for literature. He concluded his acceptance speech with the following. I
believe that man will not merely endure: he will prevail. He is immortal, not because he alone among creatures has an inexhaustible
voice, but because he has a soul, a spirit capable of compassion and
sacrifice.
The writers duty is to write about these things. It is his privilege to help man endure by lifting his heart, by reminding him of
the courage and honor and hope and pride and compassion and
pity and sacrifice which have been the glory of his past. The poets
voice need not merely be the record of man, it can be one of the
props, the pillars to help him endure and prevail.

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I hope that you Senators and this Congress find it is your privilege and your duty to fight with your intelligence and pride and
compassion to continue to build the pillars of man, the arts for the
spirit, education for the mind, and medical research for the body.
PREPARED STATEMENT

Ladies and gentlemen, let me leave you with this thought. The
stem cell is the future of medicine and I am alive because of the
progress in stem cell research. Thank you.
[The statement follows:]
PREPARED STATEMENT

OF

ANTHONY HERRERA

I was diagnosed with Mantle Cell Lymphoma in January of 1997 and was told
at New York University Hospital, This disease will kill you. There is nothing we
can do. You are going to die.
That night I debated whether to put my .38 Smith & Wesson to my temple and
pull the trigger or saddle up. I pondered each option. Then I saw myself on
HORSE heading into a dark canyon.
I found a poem by Tennessee Williams from the Night of the Iguana. I read it
every day.
I then went to Memorial Sloan-Kettering and was treated with a regimen of chop
and ICE chemotherapy and total body irradiation.
On August 1, 1997 I received an autologous stem cell transplant and was found
to be in remission.
In November 1998, I relapsed and received four cycles of chemotherapy. On March
30, 1999 I was admitted to the University of Texas, M.D. Anderson Cancer Center
in Houston Texas, under the care of Dr. Issa Khouri, M.D.
I underwent an allogeneic stem cell transplantation using a non-myeloablative
regimen. My brother John, was my donor. I required a boost of donor lymphocyte
infusion after transplantation.
I then suffered from CMV, a mild stroke and a seizure.
I was found to be in remission August 15, 1999.
This treatment was based on a concept developed at M.D. Anderson Cancer Center, that many neoplastic diseases can be treated by immune modulation only without the need for toxic high dose chemotherapy.
Up until recently high dose chemotherapy was considered essential for marrow or
stem cell transplantation.
This new treatment offers new hope and new horizons for patients suffering from
this otherwise fatal disease.
I relapsed August 15, 2000. I was told without treatment You will die in less
than twelve months. And that another donor lymphocyte infusioncould kill you.
He then worked with Dr. Ira Braunschweig, formerly of MD Anderson Cancer
Center, now medical director of Director of Bone Marrow TransplantationThe Albert Einstein College of Medicine. The plan at that time was to use Rituxan to control the lymphoma and then return to MD Anderson for a donor lymphocyte infusion.
A CAT-scan from September 27, 2000 showed less disease without any treatment
of any kind. This meant that his new immune system had started battling the disease.
This was a medical history in the treatment of Mantle Cell Lymphoma in that
the new immune system had started killing lymphoma cells and there by reducing
the amount of disease without treatment of any kind.
Dr. Braunschweig and I debated and then decided to proceed with four rounds
of high dose Rituxan during the month of October, with the hope that the Rituxan
would assist his new immune system in the battle.
CAT scans and Gallium scans that followed from November through 30 January
2001 showed a steady decrease in the amount of lymphoma and lymphoma related
activity.
Dr. Braunschweig and I have discussed several times whether there was a chance
the rituxan aided his new immune system in the battle to control the Mantle Cell
Lymphoma.
We will never know.

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What is concluded by Dr. Braunschweig, Dr. Andre Goy, Dr. James Gajewski and
Dr. Sergio Giralt is that the donor infusion of my brothers cells and the engrafting
of this new immune system in his body that has kept me in remission for five years.
This unexpected development of Graft vs. Lymphoma approach is positive news
for fighting cancer and other life threatening diseases.
Throughout this journey I listen to Willie Nelson, Louis Armstrong and Agustin
Lara of Mexico. I quoted Tennessee Williams every day. I quoted from John Fords
THE SEARCHERS . . . When was asked if he wanted to quit. John Wayne retorted, Thatll be the day.
I am alive because of great Doctors and nurses with guts and vision. 1950 William
Faulkner won the Nobel Prize for Literature . . . he concluded his speech with the
following.
I believe that man will not merely endure: he will prevail. He is immortal, not
because he alone among creatures has an inexhaustible voice, but because he has
a soul, a spirit capable of compassion and sacrifice and endurance.
The poets, the writers, duty is to write about these things. It is his privilege to
help man endure by lifting his heart, by reminding him of the courage and honor
and hope and pride and compassion and pity and sacrifice which have been the
glory of his past. The poets voice need not merely be the record of man, it can be
one of the props, the pillars to help him endure and prevail . . .
The stem cell is the future of medicine . . .
I hope you senators and this congress find that it is your privilege and duty to
fight with your intelligence and pride and compassion to continue to build the pillars of manthe arts for the spiriteducation for the mind and medical research
for the body. Stem cell research. All stem cell research.
Thank you.

Senator SPECTER. Thank you very much, Mr. Herrera, for that
very poignant and emphatic testimony and for the authentication
as to what stem cells can do, for what they have done for you.
Our next witness is Dr. Judith Gasson, Director of Jonsson Comprehensive Cancer Center at UCLA. Dr. Gasson has a doctorate in
physiology from the University of Colorado and post-doctoral work
at Saulk Institute. Thank you very much for joining us today, Dr.
Gasson, and the floor is yours.
STATEMENT OF JUDITH GASSON, Ph.D., DIRECTOR, JONSSON COMPREHENSIVE CANCER CENTER

Dr. GASSON. Thank you very much, Mr. Chairman. It is a great


pleasure to continue the conversation that you and I began several
years ago when you were visiting UCLA Medical School. At that
time, we had a very serious discussion about how important it was
that we continue to do this very important stem cell work, and I
am happy to be here today.
Cancer is now the leading cause of death in Americans under the
age of 85. This year alone, 550,000 Americans will die from their
disease. These numbers fail to account for the additional pain and
suffering felt by their families and friends.
Many scientists believe that stem cell research has the power to
revolutionize cancer therapy in much the same way that targeted
therapies have impacted cancer treatment over the past decade.
There is now considerable evidence that many types of cancer, including breast cancer, prostate, brain, and certain leukemias, arise
through mutations that occur in our adult stem cells. These socalled cancer stem cells retain the ability to self-renew, which is a
signature feature of stem cells. However, they lose the ability to respond to the proper cues and to differentiate.
Our current therapies are targeted to the bulk of the tumor and
not to the cancer stem cell. How can we develop therapies that destroy the malignant stem cells, thereby eliminating both the tumor

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and its chance to recur at a later time? Like all therapeutic advances, targeting cancer stem cells must be based on outstanding
basic science. For this reason, embryonic stem cells must be studied to educate us on the fundamental processes and pathways that
drive the growth of cancer stem cells.
To be sure, studies are ongoing on adult stem cells, but these
studies are incomplete and unable to answer all of the critical
questions. Adult stem cells are rare in our bodies and cannot be induced to grow in the laboratory without also differentiating.
We believe that characterizing the pathways that embryonic
stem cells use to self-renew, using high-throughput screening technology, will allow us to develop small molecule inhibitors to those
stem cell-specific pathways. If these chemical inhibitors of self-renewal of embryonic stem cells are isolated and characterized in the
laboratory, they may actually provide the first benefit of stem cell
research in patients.
Paradoxically, as you just heard from Mr. Herrera, bone marrow
stem cells are not only perhaps the source of some cancers, but
they also have been used to treat certain cancers for the past 4 decades. Many patients are unable to benefit from this potentially lifesaving treatment because they either do not have a matched bone
marrow donor or their own bone marrow has been compromised by
treatment or invasion of cancer cells. The technique of somatic cell
nuclear transfer would enable us to insert the DNA from a cancer
patients skin cells into an egg and reprogram that DNA to become
a pluripotent stem cell again. In this way, the patients blood and
immune systems could be reconstituted and genetically identical to
the patient.
It has been estimated that there are currently 400,000 frozen
embryos generated in in vitro fertilization clinics that will not be
used. The vast majority of these frozen cells will be destroyed.
The thousands of physicians and scientists, represented by the
American Association of Cancer Research and the American Society
of Clinical Oncology, issued public statements this year strongly
endorsing the expansion of funding for embryonic stem cell research to improve the prevention, detection, and treatment of cancer. We estimate that this represents 30,000 physicians and scientists who believe that this important work will have an impact
on the dreaded disease of cancer.
PREPARED STATEMENT

To be sure, my commitment to this area of research is professional, but it is also personal. Three years ago next week I lost my
own father to lymphoma.
Thank you very much, Mr. Chairman.
[The statement follows:]
PREPARED STATEMENT

OF

JUDITH GASSON

Cancer is now the leading cause of death in Americans under the age of 75. This
year alone 550,000 Americans will die from their disease. These numbers fail to account for the additional pain and suffering felt by their family and friends.
Many scientists believe that stem cell research has the power to revolutionize cancer therapy in much the same way that targeted therapies have impacted cancer
treatment over the past decade. There is now considerable evidence that many types
of cancer including breast, prostate, brain and leukemias arise through mutations

10
acquired in our adult stem cells. These so-called cancer stem cells retain the ability to self-renew, which is the signature feature of stem cells. However they lose the
ability to respond to normal differentiation signals.
Our current therapies are targeted to the bulk of the tumor, but not to the cancer
stem cells. How can we develop therapies that destroy the cancer stem cells, thereby
eliminating the tumor and its chances to recur? Like all therapeutic advances targeting cancer stem cells must be based upon outstanding basic science. For this reason embryonic stem cells must be studied to educate us on the fundamental processes and pathways that drive the growth of cancer stem cells. To be sure studies
are ongoing with adult stem cells, but these studies are incomplete and unable to
answer all of the critical questions. Adult stem cells are rare and cannot be induced
to grow in the laboratory without also differentiating. We believe that characterizing
the pathways that embryonic stem cells use to self-renew, using high-throughput
screens, will lead to the development of small molecule inhibitors. It is these chemical inhibitors of self-renewal of embryonic stem cells that may provide the first benefits of stem cell research in patients.
Paradoxically bone marrow stem cells have been used to treat certain cancers for
the past four decades. Many patients are unable to benefit from this potentially lifesaving treatment because they dont have a matched bone marrow donor and their
own bone marrow has been comprised by treatment or invaded by cancer cells. The
technique of somatic cell nuclear transfer would enable us to insert DNA from a
cancer patients skin cell into an egg and re-program it from a skin cell to a
pluripotent stem cell. In this way, the patients blood and immune systems could
be reconstituted and genetically identical to the patient.
Its been estimated that there are currently 400,000 frozen embryos generated
from in vitro fertilization that will not be used. The vast majority of these will be
destroyed. The thousands of physicians and scientists represented by the American
Association of Cancer Research and the American Society of Clinical Oncology
issued public statements this year strongly endorsing the expansion of funding for
embryonic stem cell research to improve the prevention, detection and treatment of
cancer.

Senator SPECTER. Thank you very much, Dr. Gasson.


Our next witness is Dr. Rudolf Jaenisch, Professor of Biology at
Massachusetts Institute of Technology and a member of the Whitehead Institute for Biomedical Research. He received his doctorate
in medicine from the University of Munich. Thank you for joining
us today, Dr. Jaenisch, and we look forward to your testimony.
STATEMENT OF RUDOLF JAENISCH, M.D., PROFESSOR OF BIOLOGY,
MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Dr. JAENISCH. Thank you, Mr. Chairman.


So I am a founding member of the Whitehead Institute and a
professor of biology at MIT. My main research interest is epigenetic
regulation, embryonic stem cells, and to understand the mechanisms of nuclear transfer and the reprogramming of the genome
following nuclear transfer. We have studied this in mice, and the
conclusion from all work was that reproductive cloning in humans
is unsafe and should be banned.
Our work was also of relevance for the therapeutic application of
somatic cell nuclear transfer. We have done this in a mouse model
again of severe combined immune deficiency, SCID. This condition
exists in humans. And we have used this technique to restore the
immune system in these mice. And I believe that this proof of principle experiment is directly relevant for treatment of human blood
diseases, such as leukemia as we heard.
The recent success by the Korean group indicates that nuclear
transfer in humans is much more efficient than we assumed before,
and they believe the treatment of bone marrow diseases will likely
be the first human disease that will be treated by SCNT.

11
Embryonic stem cells clearlyand we heard thisare of great
potential value to treat diseases, and I am confident that if we are
allowed to derive new stem cells from in vitro fertilized embryos,
that would enormously help us to understand the system. But I
want to talk about nuclear transfer today.
The proof of principle experiments are clear. In principle, this
technology will work in humans to treat diseases such as blood diseases, Parkinsons, and diabetes. We have to learn technology, but
this I think is only technology.
So what are the concerns of those who oppose nuclear cloning in
humans for the purpose of generating customized embryonic stem
cells for therapy or for research?
I believe the key concern is that the derivation of an embryonic
stem cell from a cloned construct would necessarily involve the destruction of the blastocyst and thus destruction of potential normal
human life. The crucial question is: does the cloned blastocyst really represent potential normal human life? And that is what I want
to concentrate on.
From all experience with cloned animals, I would argue that the
cloned blastocyst has little, if any, potential to ever develop into a
normal baby. Most will die in development and the few that survive to birth will develop severe defects with age because of the reprogramming faults following nuclear transplantation.
For these reasons, it has been suggested, because a cloned blastocyst is so different from the normal blastocyst which is derived
from a fertilized egg, that it should not be designated as an embryo. And I agree with this notion. However, we have to admit that
the cloned blastocyst has a chance, although an exceedingly small
chance, to develop into cloned animals such as Dolly. But Dolly
died because she suffered from major ailments, as most clones do.
But it is this statistically small chance of a clone to develop to birth
and beyond what troubles most who oppose the technology.
The altered nuclear transfer approach has been proposed by Dr.
Hurlbut as a potential solution. This approach would cause the
product of nuclear transfer to be inherently unable to ever develop
into a fetus or a baby because of its inability to establish the very
first step of embryonic organization and the inability to establish
that fetal/maternal connection. The procedure, as proposed by
Hurlbut, involves the genetic manipulation of the donor cell, not of
the embryo, with the goal to generate a construct which can still
generate embryonic stem cells but cannot implant and generate a
fetus. So the goal is, therefore, to generate what he calls a biological construct or biological artifact that lacks the essential attributes of an embryo and has no potential whatsoever to develop
into a fetus but still could proliferate and give rise to ES cells.
ANT, altered nuclear transfer, was last year proposed as a
thought experiment. We have now performed the proof of principle
experiment in the mouse, published this week in Nature, that validates this proposal. So let me explain.
In our experiment, we introduced an RNAi construct into the
skin cells prior to nuclear transfer. The RNAi was directed against
Cdx2. This is a gene which is crucial for the establishment of the
very first lineage in embryonic development which is established at
the 16-cell stage. The genetically altered skin cells do not express

12
Cdx2, but once the nucleus is transferred to the egg, the cloned
product cannot establish this key lineage. It will develop still to an
abnormal blastocyst which collapses because the trophectoderm lineage, which will give rise to the placenta, cannot form.
The embryonic stem cells derived from this construct are indistinguishable in their potential from a normal embryonic stem cell.
So the key question for the debate here is: does it generate embryos
and how abnormal are they?
So I would argue that the ANT, altered nuclear transfer, embryo
is already abnormal at the 4- to 8-cell stage molecularly because
the gene is then expressed. It is not expressed then. But it becomes
morphologically only abnormal within 2 cell divisions.
Senator SPECTER. Dr. Jaenisch, could you summarize your testimony at this point? Your full statement will be made a part of the
record.
Dr. JAENISCH. So I will then summarize that the question is can
we designate these ANT embryos as normal, these ANT blastocysts
as normal embryos. And I would think they are a mass of differentiating cells, but they definitely lack the intricate organization of
the embryo and its potential.
PREPARED STATEMENT

I want to emphasize that ANT is a modification, not an alternative, to nuclear transplantation. It requires additional manipulation of the donor cells that may complicate the logistics of an already complex procedure, and this has concerned many scientists.
However, our procedure has shown that the procedure is so simple
and straightforward that it may be acceptable as a requirement if
it would resolve the ethical objections against somatic cell nuclear
transfer and allow this research to go ahead.
[The statement follows:]
PREPARED STATEMENT

OF

DR. RUDOLF JAENISCH

Mr. Chairman and members of the Subcommittee, my name is Rudolf Jaenisch.


I am a founding Member of the Whitehead Institute and Professor of Biology at
MIT. Before coming to the Whitehead Institute I was the head of the Department
of Tumor Virology at the Heinrich-Pette Institute of the University of Hamburg in
Germany. I am privileged to have helped establish the field of transgenic science.
Transgenic science deals with the transfer of genes to create mouse models of
human disease. My present research focuses on epigenetic gene regulation, on embryonic stem cells, and on nuclear cloning. Our focus is understanding the mechanisms that bring about reprogramming of a somatic nucleus to an embryonic one
after its transfer into the egg. I work with mice and our results have demonstrated
that nuclear cloning is inefficient, that most clones die at an early embryonic stage
and that the few that survive to birth and beyond harbor serious defects and are
not normal. The conclusion from this work is that reproductive cloning of humans
is an unsafe technology that should be banned.
Our work has shown that somatic cell nuclear transfer (SCNT) can generate customized embryonic stem cells that can be used for the treatment of genetic diseases. We have performed a proof of principle experiment in mice that carry a specific mutation which causes a defective immune system. Human patients with a corresponding mutation (designated as Severe Combined Immune Deficiency or SCID)
are unable to fight infections and have a grim prognosis. In our proof of principle
experiment the nuclei of SCID mouse skin cells were transplanted into enucleated
eggs to generate cloned blastocysts (NT-blastocysts) that were then placed into tissue culture to derive customized cloned embryonic stem cells (NT-ES cells). The
genetic mutation was corrected by gene targeting, the repaired NT-ES cells were
then induced to differentiate into blood stem cells and, when transplanted back into
the mutant mouse, restored immune function. I believe that this proof of principle

13
experiment is directly relevant for the treatment of human blood diseases such as
thalassemia, sickle cell anemia or leukemia. The recent success by the Korean group
(Hwang et al.) indicates that nuclear transfer in humans is more efficient than was
assumed before and I believe that treatment of bone marrow diseases will likely be
one of the first human diseases that will be treated with SCNT.
Embryonic stem cells have an enormous potential for therapy of debilitating diseases such as cancer, diabetes, Parkinsons or other degenerative diseases. To realize this therapeutic potential much research is needed to learn how to differentiate
the embryonic cells into cells used for transplantation. I am confident that the possibility to derive new ES cell lines from IVF embryos as debated in Congress would
enormously help this research.
I will focus on nuclear transfer (NT). In addition to its potential for customized
therapy, nuclear transfer derived ES cells would be an extraordinary important tool
to study complex diseases such as ALS or Alzheimers in the test tube since customized ES cells derived from a patient would carry all the genetic alterations that
caused the disease in the patient. The exciting prospect is that differentiation of the
ES cells in the culture dish may provide clues to what goes wrong with the cells
and how to establish therapies. This is not a future promise but this could be done
today using the technology established by the Korean group that was the first to
successfully derive human stem cells from cloned blastocysts.
What are the concerns of those who oppose nuclear cloning in humans for the purpose of generating customized embryonic stem cells for research or for therapy?
I believe the key concern is that the derivation of an embryonic stem cell would necessarily involve the destruction of the blastocyst and thus the destruction of potential human life. The crucial question is: does the cloned blastocyst really represent
potential normal human life?
From all experience with cloned animals I would argue that the cloned blastocyst
has little if any potential to develop into a normal baby as most would die in development and the few that survive will be abnormal and will develop severe defects
with age. This is because reprogramming of the somatic cells genome after nuclear
transplantation is a faulty process causing the great majority of clones to have hundreds of genes incorrectly expressed. For these reasons it has been suggested that,
because the cloned blastocyst is so different from the normal blastocyst derived from
a fertilized egg, it should not be designated as an embryoand I agree with this
notion. However, the cloned blastocyst has some chance, an exceedingly small
chance, to ever develop into a cloned animal such as Dolly. And Dolly died early
because she suffered from major ailments due to faulty reprogramming as most if
not all cloned animals do. It is this statistically small chance of a clone to develop
to birth and beyond that troubles, I believe, those who are opposed to the NT technology.
The Altered Nuclear Transfer (ANT) approach has been proposed by Dr. Hurlbut
from Stanford as a potential solution for the ethical dilemma. This approach would
cause the product of nuclear transfer to be inherently unable to ever develop into
a fetus or a baby, because of its inability to establish the very first step of embryonic
organization and its inability to establish a fetal-maternal connection. With other
words, the ANT procedure would reduce the statistically low chance of an NT blastocyst to develop to birth to zero. The procedure, as proposed by Hurlbut, involves
the genetic manipulation of the donor skin cell with the goal to inactivate a gene
that is required for embryo development if the nucleus of the manipulated cell
would be transplanted into an enucleated egg as in SCNT. The manipulation would,
however, have no ill effect on the derivation of embryonic stem cells from the product of SCNT. Thus, the alteration causes the somatic nucleus to function in such
a way that no embryo is generated but embryonic stem cells can be produced. The
goal of ANT is to generate a nuclear transfer product that lacks the essential attributes of an embryo and has no potential whatsoever to develop into a fetus but
still could proliferate and give rise to embryonic stem cells. ANT was suggested last
year as a thought experiment. We have now performed a proof of principle experiment in the mouse (published this week in Nature) that validates the proposal.
In our experiment an RNAi construct that inactivates the Cdx2 gene was introduced into skin cells. Cdx2 has a crucial function in the establishment of the first
embryonic lineage, the trophectoderm that is established at the 16-cell stage and
forms the placenta of the embryo. Skin cells normally do not express the Cdx2 gene.
But when used as donors for nuclear transplantation, the ANT product is unable
to activate the gene and therefore unable to establish the trophectoderm lineage.
However, the product of nuclear transfer did proliferate and formed an abnormal
NT-blastocyst. The normal blastocyst consists of the inner cell mass (which will form
the embryo proper) and a cavity which is surrounded by trophectoderm cells (which
will form part of the placenta). In contrast to the normal embryo, the ANT blasto-

14
cyst collapses because the trophectoderm cells are lacking. Importantly, when placed
into tissue culture, the ANT blastocyst generates embryonic stem cells that have the
full potential for differentiation and therapy and thus are indistinguishable from
embryonic stem cells that are derived from a fertilized embryo.
Does the ANT procedure generate embryos, even if only abnormal ones? Our experiments clearly show that the Cdx2 deficient blastocyst has no potential to implant and to ever develop into a fetus because it lacks the trophectoderm lineage
that gives rise to the placenta. Cdx2 is activated at the 8-cell stage and activation
of this key gene is prevented in the ANT product. Thus, the product of ANT-SCNT
is already molecularly different from the normal embryo at the 8-cell stage and becomes morphologically abnormal within the next two cell divisions. The placenta is
an integral part of the embryo and not some component that could be separated
from the embryo. It is like the engine of a car: one cannot separate the engine from
the car and still call it a car. Because the ANT product lacks essential properties
of the fertilized embryo, it is not justified to call it an embryo.
It is important to emphasize that ANT is not an alternative to nuclear transplantation but a modification of an experimentally highly demanding process. It requires
additional manipulations of the donor cells that will complicate the logistics of an
already complex procedure, and this has raised concerns among many scientists.
Also, it has not been determined whether Cdx2 has a similar function on human
placentation as in mouse. Because the effect of gene inhibition on human placentation cannot be directly tested, surrogate assays such as in vitro differentiation of
human ES cells are required to assess the effect of CDX2 deficiency on human placental development. The experiments in mice have shown a proof of concept of the
ANT procedure. It would be unfortunate, however, if the implementation of this approach would delay the research on human SCNT.

Senator SPECTER. Thank you very much, Dr. Jaenisch.


Our next witness is Dr. Steven Teitelbaum, Professor of Pathology at Washington University School of Medicine, an M.D. from
Washington University, residency at New York University. Thank
you very much for coming in today, Dr. Teitelbaum, and we are interested in hearing your testimony.
STATEMENT OF STEVEN TEITELBAUM, M.D., WILMA AND ROSWELL
MESSING, PROFESSOR OF PATHOLOGY AND IMMUNOLOGY,
WASHINGTON UNIVERSITY SCHOOL OF MEDICINE

Dr. TEITELBAUM. Mr. Chairman, I thank the committee for the


honor of speaking to you today.
Mr. Chairman, I have been a physician scientist for more than
30 years. I have authored in excess of 300 scientific papers, and I
am here to tell you that in my estimation we are facing a unique
opportunity in the form of embryonic stem cell research to potentially alleviate the misery of our fellow Americans with a number
of presently incurable diseases. But to get there, we must do the
science.
Opponents of embryonic stem cell research often articulate their
position as a contest between adult and embryonic stem cells. Mr.
Chairman, this is not a contest between various types of stem cells.
It is a contest between us as a society and disease. We should be
moving forward on all fronts, adult, embryonic, and umbilical cord
stem cells to win the battle. The tool is not important. What counts
is curing our neighbors.
That said, because of their flexibility, embryonic stem cells hold
more promise to ameliorate presently incurable diseases than any
other approach. I stress the word promise because we are not
there yet, and it is my belief that it will be some time before we
are positioned to safely use these cells for therapy. But if scientists
are prevented from exploring the biology of human embryonic stem
cells, we will never get there.

15
Mr. Chairman, as you know, human embryonic stem cells can
presently be obtained from two sources; namely, the spare products
of in vitro fertilization, which ultimately would be destroyed, and
by somatic cell nuclear transfer, also known as SCNT or therapeutic cloning. Although both approaches hold enormous therapeutic potential, I am particularly taken with the promise of SCNT
because it may alleviate the major complication of tissue and cell
transplantation, namely rejection and its attendant life-threatening
consequences.
Mr. Chairman, I am a bone biologist and physician, and as such,
I see many patients who have received organ and cell transplants.
These patients typically develop severe osteoporosis and often have
many fractures because of the harsh medications they must take
to prevent rejection of their transplant. It is my hope that embryonic stem cells, generated by SCNT, which contain the transplant
recipients own DNA will reduce the necessity for these devastating
anti-rejection drugs.
But, Mr. Chairman, my hopes for SCNT are more personal and
harken back more than 20 years when I was a young assistant professor. At that time, I became interested in a genetic disease of the
skeleton known as osteopetrosis, or marble bone disease, and I
want to tell you a story about a child who profoundly impacted my
life.
Osteopetrosis is a disease in which kids make too much bone.
Consequently, their skulls become very thick and compress their
brains and nerves, such as those leading to the eye. Bone also overgrows the bone marrow, preventing formation of blood cells. Until
the story I am about to tell you, all kids with the malignant form
of osteopetrosis developed fatal neurological complications, including blindness, and infections due to bone marrow suppression.
These children invariably died in the first decade, most before the
age of 5.
In the early 1980s, our team thought we had identified the abnormal cell causing osteopetrosis and concluded it resided in the
bone marrow. We reasoned, therefore, that if we gave an
osteopetrotic infant a bone marrow transplant which contains adult
stem cells, we might cure the disease. We realized the enormous
risk of rejection, so we waited until we had a perfect immunological
match between the donor and recipient, in this instance the 3month-old little girl you see in the top picture. So we gave this
baby a bone marrow transplant and achieved the first cure of this
disease. The middle panel shows her at 3 years of age, and the bottom picture, which is recent, was taken upon her graduation from
college. Senators, being part of a team which was first to cure a
fatal disease, particularly that of children, is a doctors dream. It
does not get any better.
You may be asking yourselves why this guy, who is here as an
advocate of embryonic stem cell research, is telling us about his
victory with adult stem cells. Senators, I am recounting the story
to underscore the importance of moving forward on all fronts because, regrettably, there is a down side to my tale. You will remember that this was a perfect immunologic match, and therefore there
was little chance of rejection. Unfortunately, such matches are extremely rare and therefore, we presently cure less than 10 percent

16
of kids with osteopetrosis. The use of SCNT, in which embryonic
stem cells contain the patients own DNA, if successful, would
markedly increase the cure rate of this disease.
PREPARED STATEMENT

Mr. Chairman, because of my familiarity with osteopetrosis, I am


frequently contacted by parents with afflicted children. I have to
tell them that the chances of curing your child is no more than 10
percent. I want to tell them it is greater than 90 percent. SCNT,
if we pursue it, may get us there.
Thank you.
[The statement follows:]
PREPARED STATEMENT

OF

DR. STEVEN TEITELBAUM

Thank you Mr. Chairman. My name is Steven Teitelbaum. Im the Wilma and
Roswell Messing Professor of Pathology and Immunology at Washington University
School of Medicine and I thank the committee for the honor of speaking to you
today.
Mr. Chairman, Ive been a physician-scientist for more than 30 years. Ive authored in excess of 300 scientific papers and Im here to tell you that, in my estimation, we are facing a unique opportunity in the form of embryonic stem cell research, to potentially alleviate the misery of our fellow Americans with a number
of presently incurable diseases. But to get there, we must do the science.
Opponents of human embryonic stem cell research often articulate their position
as a contest between adult an embryonic stem cells. Mr. Chairman, this is not a
contest between various types of stem cells. It is a contest between us as a society
and disease. We should be moving forward on all fronts, adult, embryonic and umbilical cord stem cells, to win the battle. The tool is not important. What counts is
curing our neighbors.
That said, because of their flexibility, embryonic stem cells hold more promise to
ameliorate presently incurable diseases than any other approach. I stress the word
promise because we are not there yet and it is my belief that it will be some time
before we are positioned to safely use these cells for therapy. But if scientists are
prevented from exploring the biology of human embryonic stem cells, we will never
get there.
Mr. Chairman, as you know, human embryonic stem cells can presently be obtained from two sources, namely the spare products of in vitro fertilization, which
ultimately would be destroyed, and by somatic cell nuclear transfer, also known as
SCNT or therapeutic cloning. Although both approaches hold enormous therapeutic
potential, Im particularly taken with the promise of SCNT because it may alleviate
the major complication of tissue and cell transplantation, namely rejection and its
attendant life threatening consequences.
Mr. Chairman, Im a bone biologist and physician and as such I see many patients
who have received organ and cell transplants. These patients typically develop severe osteoporosis and often have many fractures because of the harsh medications
they must take to prevent rejection of their transplant. It is my hope that embryonic
stem cells, generated by SCNT, which contain the transplant recipients own DNA,
will reduce the necessity for these devastating anti-rejection drugs.
But Mr. Chairman, my hopes for SCNT are more personal and hearken back more
than 20 years when I was a young assistant professor. At that time I became interested in a genetic disease of the skeleton known as osteopetrosis or marble bone disease and I want to tell you a story about an afflicted child who profoundly impacted
my life. Osteopetrosis is a disease in which kids make too much bone. Consequently,
their skulls become very thick and compress their brains and nerves, such as those
leading to the eye. Bone also overgrows the bone marrow preventing formation of
blood cells. Until the story Im about to tell you, all kids with the malignant form
of osteopetrosis developed fatal neurological complications, including blindness, and
infections due to bone marrow suppression. These children invariably died in the
first decade, most before the age of five.
In the early 80s, our team thought we had identified the abnormal cell causing
osteopetrosis and concluded it resided in the bone marrow. We reasoned, therefore,
that if we gave an osteopetrotic infant a bone marrow transplant, which contains
adult stem cells, we might cure the disease. We realized the enormous risk of rejection so we waited until we had a perfect immunological match between the donor

17
and recipient, in this case a 3 month old little girl you see in the top picture. So
we gave this baby a bone marrow transplant and achieved the first cure of this disease. The middle panel shows her at 3 years of age and the bottom picture, which
is recent, was taken upon her graduation from college. Senators, being part of a
team which is first to cure a fatal disease, particularly of children, is a doctors
dream. It doesnt get any better.
You may be asking yourselves why this guy, who is here as an advocate of embryonic stem cell research, is telling us about his victory with adult stem cells. Senators, Im recounting the story to underscore the importance of moving forward on
all fronts because regrettably there is a downside to my tale. Youll remember that
this was a perfect immunological match and therefore there was little chance of rejection. Unfortunately, such matches are extremely rare and therefore we presently
cure less than 10 percent of kids with osteopetrosis. The use of SCNT, in which embryonic stem cells contain the patients own DNA, if successful, would markedly increase the cure rate of this disease. Mr. Chairman, because of my familiarity with
osteopetrosis Im frequently contacted by parents with afflicted children. I have to
tell them the chances of curing your child is no more than 10 percent. I want to
tell them its greater than 90 percent. SCNT, if we pursue it, may get us there.

Senator SPECTER. Thank you very much, Dr. Teitelbaum.


Our final witness is Dr. John Wagner, Professor of Pediatrics and
Scientific Director of the Stem Cell Institute at the University of
Minnesota. An M.D. at Jefferson Medical College in Philadelphia
and internship and residency at Duke University School of Medicine. Thank you for coming to Washington today, Dr. Wagner, and
we look forward to your testimony.
STATEMENT OF JOHN WAGNER, M.D., SCIENTIFIC DIRECTOR OF CLINICAL RESEARCH, BLOOD AND MARROW TRANSPLANT PROGRAM
AND STEM CELL INSTITUTE

Dr. WAGNER. Mr. Chairman and committee members, I am coming here as a clinician, as a stem cell researcher. It is not a question of whether or not this knowledge is going to be translated into
something clinically useful. The real question is, when is that going
to happen?
The work should not be restricted to private industry. Stem cell
research should be taking place in academic institutions, supported
by Federal dollars with guaranteed oversight, peer review, and
transparency.
Right now, as we have heard already in testimony this morning,
there is only one proven use of stem cells and that is in the context
of blood and marrow transplantation to treat diseases like leukemia, lymphoma, sickle cell disease, and a variety of other blood
and immune disorders. In these instances, we need to infuse stem
cells to repair the marrow that has been destroyed either by the
disease itself or by the therapy we use to treat that disease, such
as with chemotherapy and irradiation. These blood-producing stem
cells are found in marrow and they are found in cord blood, which
is the blood that is left in the placenta after a baby is born.
Tremendous achievements have already been made in these
areas, particularly in the area of cord blood most recently, and in
fact, the Institute of Medicine last April made recommendations
that we significantly augment the Nations inventory of cord blood
to help take care of our patients around the country.
While my own work is focused on the development of stem cell
therapies from cord blood or adult tissues and, perhaps surprisingly, not embryonic stem cells, I am here today really to defend
ES cell work. It must be unequivocally clear that our work in cord
blood and adult stem cells does not eliminate the need for work in

18
ES cells. Yes, it is true that stem cells and cord blood and adult
tissues can differentiate into perhaps the lining cells of the gut or
the liver or neural tissue, but they do not exhibit all the capacities
of ES cells. For example, we have yet to see stem cells from cord
blood or adult tissues differentiate into heart muscle that spontaneously beats in the petri dish. That has been shown repetitively by
people working on ES cells.
The University of Minnesota is well known for its work in adult
stem cells in umbilical cord blood, and with Catherine Verfaille, we
have pioneered that work in cord blood and multipotent adult stem
cells and we see great promise in those areas. But we recognize,
although there is tremendous potential, there are also limitations.
It is critical that you also know that every discovery that has occurred with ES cells has really benefitted us working on adult stem
cells and cord blood.
But speaking as a clinician who creates these new stem cell
therapies for treating children and adults with a variety of incurable diseases, it not only gives us significant hope, but it also
comes with risk. This winter we hope to be able to try our first
stem cell transplants in the treatment of patients damaged by
chemotherapy and irradiation, not just for bone marrow recovery,
but also the other tissues that are involved in the treatment and
damaged by it. We have to go through the ethics committees, we
have to go through human subjects committee, and the FDA. But
we are going to move this therapy forward, obvious, with all the
proper oversight.
But it is incomprehensible that we do otherwise, that we restrict
ourselves to one type of stem cell. Like others in this room, I feel
compelled to move this forward on behalf of the thousands of patients that write to me every week asking to allow them to be the
first stem cell recipient. In fact, this is just one e-mail that I received yesterday from a woman who is 39 years old saying, I had
a stroke several years ago. What can you do for me? Let me be the
first. Why can I not be a healthy wife to my husband, a mother
to my young child?
Is this all hype? Where are the first trials with ES cells? Certainly the lack of funding and restricted access to suitable stem cell
lines has been a major barrier in our research efforts. We need to
address those barriers where possible. For example, can we separate reproductive cloning from nuclear transfer? If you desire rapid
translation of ES cells into real clinical therapies, let us not restrict
it. We need to be able to use nuclear transfer because it is likely
to be instrumental in moving that therapy forward as quickly as
possible.
Again, this is not some scientists dream. It has been done with
human cells, at least in South Korea. Every single one of us will
be faced with a disease amenable to stem cell therapy. It may be
our child, our spouse, our friend, or even ourselves, and you can
ask Mr. Herrera and you can ask Ms. Carolyn Kohn, who is in the
audience, who had a child die of aplastic anemia.
Cord blood certainly has its proven benefits in the treatment of
a variety of diseases. It has great potential perhaps in the future
for tissue repair that yet has clearly to be identified. Federal dol-

19
lars should be devoted to the work of all these stem cell sources,
including ES cells. ES cell work must continue in parallel.
PREPARED STATEMENT

As a clinician that treats these patients who are defined as incurable, I feel obligated to be here on their behalf. I am sure that
many of them are anxiously waiting to hear what happens today.
For them, the stakes must be simply unimaginable.
[The statement follows:]
PREPARED STATEMENT

OF

DR. JOHN E. WAGNER

Stem cell therapy will revolutionize the practice of medicine. For the first time
there will be treatments for spinal cord injury, diabetes, cancer, stroke, and heart
disease with potentially long term benefits. The proof of principle already exists.
It is not a question of whether this new knowledge will translate into clinical
therapies but rather how long. Will clinical trials in diabetes or stroke be soon or
decades away? Will this work be driven by private industry without any oversight
or in academic environments using federal support; conducted in university settings
which guarantee requisite oversight, publication, peer review and transparency?
So what do we know about stem cells today?
There is only one proven established use of stem cells and that is in the setting
of bone marrow transplantation. For decades it has been known that marrow stem
cells can be transplanted from one individual to another in order to replace the
blood and marrow of patients with leukemia/lymphoma/multiple myeloma/other diseases after their own marrow has been destroyed by disease or treatment with high
doses of chemotherapy and radiation. These stem cells come from adult marrow or
umbilical cord blood.
My own work is focused on umbilical cord blood and development of novel phase
I clinical trials. In this discussion, we cannot forget that cord blood is already an
established treatment with tremendous potential. Recently, the Institute of Medicine summarized its findings on the benefits of cord blood and the urgent need to
expand the useable inventory. Cord blood is rapidly becoming the standard of care
in children. We have recently reported outcomes in adults with results that are unprecedented. However, it must be clear that cord blood stem cells are not the stem
cells found in embryonic stem cell lines. The stem cells in adult tissues and umbilical cord blood have different properties and may or may not have unlimited differentiation capacity. While it is hoped that one day we will be able to take adult
tissue or cord blood stem cells and trick it to become ES-like, this is not yet possible. Despite what the opponents to ES cell work would suggest, it is simply not
true.
The University of Minnesota is well known in the field of stem cell research. We
have the longest standing Stem Cell Institute in the country. My work in umbilical
cord blood stem cell research and Catherine Verfaillies work on the multipotent
adult stem cell clearly demonstrate our hope to maximize the potential of cord blood
and adult tissue stem cells but we recognize that there are limitations. Of course
we are excited about the future potential of these stem cells but never have we suggested that they obviate the need for ES cell research. For example, never have the
stem cells from cord blood or adult tissues ever produced heart muscle cells that
spontaneously beat or formed islets that secrete insulin, as has been shown repeatedly with ES.
It is critical for the public to know that if we are ever to make cord blood and
adult tissue stem cells function like ES cells, we need to study ES cells. Every discovery with ES cells has furthered our work with stem cells from umbilical cord
blood or adult tissues.
Now speaking as a clinician who actually performs new therapies with stems cells
in humans, we are indeed planning to perform the first clinical trial with multipotent adult stem cells this winter in an attempt to repair tissues damaged by radiation and chemotherapy. My goal is to move stem cell therapy forward in numerous
areas as the clinical director of the Stem Cell Institute. Once we meet the requirements of the Human Subjects Committee, FDA, Ethics committees, we plan to move
stem cell therapies forward regardless of whether they are ES, cord blood or adult
tissue-derived. It is incomprehensible to do otherwise. Like others, I receive thousands of letters, emails, phone calls per month asking me to allow them to be the
first to receive stem cell treatmentsthese people have cancer, spinal cord injury,

20
diabetes, strokes, Parkinsons disease, and other genetic diseases. (Show sample
emails from this week).
You ask, what is the future of ES cells to cure a diseasethe answer is simply
breathtaking. Clearly there are risks as ES cells if left undifferentiated have a
propensity to cause tumors. But still, many are working to make these cells therapeutically valuable. In addition to the development of novel strategies for treating
Parkinsons, diabetes, stroke and spinal cord injury, some like Daniel Kaufman at
the University of Minnesota are focused on manufacturing red blood cells in massive
scale thus reducing our dependence upon volunteer donors or developing nature killer cells as anticancer agents-both derived from ES cells. So why has there not been
a single trial thus far with ES cellfunding, access to suitable cells lines, and research on the immune response to these stem cells. Nuclear transfer will be crucial
to this successtailor made stem cells lines for individuals will be required to
counter likely immune responses. Again, this is not futuristic, the South Korean scientists have clearly demonstrated that this is not just desirable but possible.
To restrict work with ES cells or bar SCNT would cripple our capacity to move
all stem cell therapies forward ES cells are the gold standard and research with
them will maximize the potential of cord blood and adult stem cells and pursuit of
multiple approaches will permit the most rapid translation of stem cells possible
into efficacious clinical therapies. Every single one of us will be faced with a child,
friend, loved one, or even ourselves with a disease amenable to stem cell therapy
in the not too distance future. Umbilical cord blood has proven benefits in the treatment of leukemia, lymphoma, blood disorders, immune deficiencies and metabolic
diseases today. Banking of cord blood is in the nations interest and federal dollars
should continue to be spent to determine the breadth of what it can offer well beyond the confines of blood and marrow diseases. At the same time in parallel, we
must also push ES and adult stem cells to the limits of what they can offer. And
for ES cells, banning SCNT could prevent its future success as SCNT is likely to
be the key that will make ES cell therapies more widely available more rapidly. I
am here as an advocate for the thousands of people who have asked me to push
this forward.

Senator SPECTER. Thank you very much, Dr. Wagner.


Mr. Herrera, you have testified about your situation being a
medical breakthrough in medical history. Was the aspect of using
stem cells on your lymphoma the unique breakthrough that you referred to?
Mr. HERRERA. Without question. The difference between the first
transplant and the second transplant was at the first transplant,
they took stem cells out of my body. I injected myself with a drug
called Neupogen every day. This causes the bone marrow to overproduce. Little baby stem cells are floating around in the blood.
They stick a pipe in here, run it through a machine, and they take
out the little baby stem cells.
The problem with that transplant for mantle cell lymphoma,
which they were not aware of at the time, was this is my immune
system.
So the theory of the allogeneic stem cell transplantI go back to
my Mississippi rootswe are having civil disorder in Wiggins, Mississippi, so we call out the Stone County National Guard. I am told
not to let anybody cross this line. Winfield Alexander wants to
cross the line. I cannot stick Winfield in the gut with a bayonet because he was my Boy Scout leader in the rattlesnake patrol. But
if you bring in the National Guard from Montana, they speak the
same language, they can read the signs, and they are not going to
have a problem sticking Winfield with a bayonet. So the foreign immune system is going to be tougher on the lymphoma, on the blood
cancer, because it does not know it that well. That is kind of a
basic comment, but that is how I had to understand it.
Senator SPECTER. Thank you, Mr. Herrera.

21
I very much regret that I am going to have excuse myself at this
point. I turn the gavel over to Senator Cochran.
What I would appreciate your doing, each of you, is to write a
memo or a letter to the subcommittee as to what you could do if
Federal funding were available for your stem cell research. Dr.
Gasson is from UCLA where I visited several years ago. Without
the particulars at hand, I know UCLA is the beneficiary of very
substantial NIH grants.
This subcommittee, Senator Harkin, Senator Cochran, and then
the full committee has taken the lead in increasing Federal funding
from $12 billion to $28 billion. And we are now on the cutting edge.
Dr. Wagner, you talked about use of Federal funds.
I regret that there are not more Senators available, but this is
the third time it will be said. This is a very, very busy place, but
your testimony is transcribed. Staff are here and Senators will review it. If you would supplement what you have testified to, Dr.
Teitelbaum, Dr. Jaenisch, Dr. Wagner, Dr. Gasson, with what the
Federal funding could do. We are going to have a vote on this one
day soon, without going into all the technicalities. And the evidence
that you will present will be very helpful when we fight it out on
the Senate floor. Things are quiet here today, but we are going to
have a pretty heavy debate on this subject and your participation
and your evidence will be very, very helpful in achieving a very,
very important goal for medical science.
Senator Cochran, let me thank you for taking the gavel. It belongs to you anyway.
Senator COCHRAN [presiding]. Thank you for your patience with
our change of command and responsibility.
I appreciate so much each of your efforts to be here today, to
take time to prepare a presentation for our committee so that we
can better understand the challenges and the responsibilities that
we have for identifying ways that we can continue to support medical research, to take those actions that will help find cures for diseases, prevent diseases. So this is all very serious business, and I
appreciate very much the fact that you have taken time and devoted your efforts and energies to this hearing today.
Senator Specter, as I said in my opening comments, has been a
champion for medical research, and the figures that he cites, the
increase in the funding that we have been able to provide or to recommendwe do not get to decide. We recommend to the full committee. The full committee approves and recommends to the Senate, and we have to work out differences between the Senate and
the House. But it has been a successful campaign to more than
double the amount of money that is available for researchers and
those providing treatment in our battle to find cures and to prevent
disease, particularly cancer.
Let me ask a few questions. I understand, Dr. Teitelbaum, you
are at the Washington University School of Medicine and have
completed a residency at New York University. Let me ask you.
What would you say is the overwhelming opinion of scientists regarding the need to expand the current stem cell policy? Is there
any disagreement within the community?
Dr. TEITELBAUM. I think not, Senator. I think that the overwhelming opinion of scientists is to move forward on all fronts, that

22
there is potential in adult stem cell research, embryonic stem cell
research, and umbilical cord blood stem cell research. We cannot
determine which technique will yield what results until we do the
science.
Senator COCHRAN. Dr. Gasson, I heard your comments, before I
had to step out of the hearing room, in your opening statements.
What forms of cancer do you think will be the most responsive to
drugs developed using stem cells?
Dr. GASSON. We believe that those types of cancer that have been
shown experimentally to be derived from a mutated adult stem cell
would be the very best targets for those small molecules. Fortunately or unfortunately for us, they include some of the most common forms of cancer, such as breast cancer, prostate, colon, some
of the leukemias, and as you are probably quite well aware, brain
tumors which are truly devastating.
We think that the concept of the cancer stem cell explains a lot
about the natural history of the disease. The patient develops cancer and is treated with surgery, radiation, perhaps chemotherapy,
and the bulk of the tumor, the large mass of the tumor goes away.
But for some patients over the next 2, 3, 4, or 5 years, the tumor
comes back and the tumor that recurs is typically even more aggressive. And we think this is because the treatments that we have
now do not kill the tumor stem cell, and so slowly it begins to divide and it recreates the tumor cells in the patient. And now those
cells are even more resistant to the treatments that we have. So
until and unless we are able to either destroy the cancer stem cell,
or at least keep it under control, we will be continuing to face the
possibility of recurrence in these very common and very deadly
cancers.
Senator COCHRAN. Dr. Jaenisch, there was some indication in a
Washington Post article that the altered nuclear transplantation
technique that your lab has achieved may be a way around the objections of some who oppose embryonic stem cell research. Would
you have pursued this line of research if not for the restrictions in
place on stem cell research?
Dr. JAENISCH. I think our research had two goals. One is a scientific one. We wanted to see whether these cells can do what we
thought they could do. But I think the major goal was to find a potential compromise which could compromise between the concerns
of those who object to the nuclear transfer procedure and those who
think that is really important to do.
So I think the altered nuclear transfer procedure is a modification of the nuclear transfer procedure. It is an additional step
which complicates an already complex procedure. But from our experience with mice at least, it is such a straightforward and simple
modification that it may be acceptable as a compromise if that
would allow then this research to go on and to provide the funding
for this type of research.
Senator COCHRAN. Is the kind of research you are doing susceptible to funding by the National Institutes of Health?
Dr. JAENISCH. All my research is funded by the National Institutes of Health, but I work with mice, so it is not controversial. But
we would like to move into human cells. We would like to work
with the new human stem cells. We would like to understand how

23
the human cells compare with the mouse cells, and we are very
limited. We do not have funding for that.
Senator COCHRAN. Well, do you think that Federal funding
should be diverted from other forms of stem cell research to support alternative methods to derive stem cells?
Dr. JAENISCH. Well, I am not sure if it should be diverted. As
was said by all the speakers here, we really need to pursue all
these avenues. So alternative methodsseveral have been proposed.
I think the final goal of the field in my opinion is to understand
what reprogramming means. How does the egg reprogram a somatic nucleus and eventually do it without the egg. In order to get
there, we need the egg. We need human eggs to learn how the
human egg does reprogramming. So I think it is counterproductive
for this goal at this point that research is not allowed to use
human eggs.
Senator COCHRAN. Anthony, I am so pleased that you were able
to be here today to put in perspective from a patients point of view
how important research in the development of new treatments,
finding new ways of dealing with these medical problems will be,
and the role that we can play. I know if you had a vote, you would
probably vote to quadruple, double, exponentially increase funding.
But where in the area of research from your experience do we
need to supplement and try to provide more incentives through
Federal research appropriations to achieve the goals of curing cancers like yours?
Mr. HERRERA. What these ladies and gentlemen up here have
said is that there should be massive amounts, billions of dollars,
and no restrictions on any of this research because I have met with
the doctor at M.D. AndersonAndreyev I believe is his nameand
we were talking about the embryonic versus the adult. He said we
need lots of room to experiment.
The drug that helped save my life, which has probably saved
hundreds of thousands of lives, Neupogen, was developed by Janice
Gabrilove and two other doctors. She was in charge of my first
bone marrow transplant. I said, how did you develop this drug?
She said we did not have a straight line. We were in there in that
region working, and all of a sudden there was a path we could follow.
So there needs to be no restriction. There needs to be massive
amounts of money. South Korea, Singapore 2 years ago were ahead
of us. China just put billions of dollars into research. So there
should be no restrictions and massive amounts of money put behind this in my opinion.
Senator COCHRAN. From your experience, could you tell us in
your own words what the difference is from a patients point of
view in a bone marrow transplant therapy and a stem cell transplant therapy?
Mr. HERRERA. Actuallysomeone please correct me if I am
wrongthey are the same thing except the way you get the cell.
The reason it is called a bone marrow transplant is before this drug
Neupogen and before the apheresis machine, they had to drill into
the bone marrow to suck out marrow and then get the stem cell
out of that. Am I correct on that? So it evolved into simply being

24
called the stem cell transplant. Some hospitals still drill into the
bone and suck out the marrow to get the stem cell, but they are
ultimately the same thing.
Senator COCHRAN. It does not sound like much fun.
Mr. HERRERA. There was not a lot of fun through the whole process, Senator.
Senator COCHRAN. I can remember reading your description of
the pain that you suffered in that first effort to get some of your
bone marrow. No anesthetic.
Mr. HERRERA. That was not good medicine.
Senator COCHRAN. I hope that is not a widespread practice now.
Mr. HERRERA. I have learned that it is not.
Senator COCHRAN. Good.
Dr. Wagner, we appreciate your being here as well. Since your
primary interest appears to be cord blood stem cell research, as I
understand it, why are you so supportive of embryonic stem cell
and nuclear transplantation research?
Dr. WAGNER. My interest is, obviously, in cord blood as one avenue. As the clinical director of the Stem Cell Institute, I am really
interested in all aspects of stem cell therapies, whether it comes
from embryonic stem cells, adult tissues, or umbilical cord blood.
So we are exploring all avenues.
However, my own personal research area in the laboratory is
with umbilical cord blood and trying to figure out what really the
breadth of applicability will be. So we are investigating not only in
the context of classical bone marrow transplantation, which has
proven to be of great use, but also looking at what its differentiation potential is, can it differentiate into various tissues.
But remember that what we said over and over again is that ES
cells are the gold standard by which everything is compared. And
everything that we have learned with embryonic stem cells, in
terms of the mechanisms of what makes them able to become liver
or lung or brain, or whatever it is, has given us clues or techniques
that allow us to see if we can get adult tissues or cord blood tissues
to do the same thing. So without having that research move forward with embryonic stem cells, we have no hope to make adult
tissues or cord blood stem cells become you would all like it to become as the stem cell source.
Senator COCHRAN. Does it surprise you that the NCI funds less
than $5 million worth of embryonic stem cell research? And why
do you think the level of stem cell research is so low in the context
of a $5 billion budget?
Dr. WAGNER. You are asking my opinion now.
Senator COCHRAN. Yes.
Dr. WAGNER. Well, clearly, I think the reason why the budget is
so low is, in part, related to the ethical issues associated with embryonic stem cell work. However, there is considerable funding for
adult tissue stem cells, as well as umbilical cord blood. However,
what we need to be doing is working on embryonic stem cells. Unfortunately, the budget is low and it has actually been extraordinarily restrictive in what we are able to do.
Right now as the clinician that hopes to move some of these cell
therapies forward, we have no hope of using the existing stem cell
lines that are currently approved because of the fact that many of

25
them were developed on murine feeder layers or they have cytogenetic abnormalities having been passaged in a culture. And as
someone who manufactures cells for clinical use, they would never
fulfill our criteria. So certainly we need to markedly expand the
amount of resources or else we will never be able to move it forward.
Senator COCHRAN. Dr. Gasson, you also lead an NCI-designated
comprehensive cancer center and support research on embryonic
stem cells. Do you have an opinion about the disparity in terms of
the $5 million for stem cell research compared with a $5 billion
budget?
Dr. GASSON. I have two additional thoughts to add to Dr. Wagners comments.
First of all, this notion that cancer arises from a cancer stem cell
is fairly new. If you are trying to study the cancer stem cell, that
is an adult stem cell, and so that particular type of work has just
recently been done and probably would not be counted under the
rubric of embryonic stem cell research.
But the main reason is the reason that Dr. Jaenisch articulated,
which is most of the people that are trying to work in this field are
working with mouse ES cells and mouse models because of the restrictions on the use of human ES cells. And Dr. Jaenisch is a perfect example. These people are extraordinarily talented. They have
devoted their careers to understanding these things. If we could
channel them from the mouse to working on human ES cells, we
could accelerate the pace of progress enormously. So it is a followup on Dr. Wagners answer, which is that the restrictions are pushing people to work in the mouse system.
CONCLUSION OF HEARING

Senator COCHRAN. Let me thank all of you for your generous contribution of time and effort to this hearing. We appreciate it very
much, and I am sure we will benefit from your observations and
your wisdom as we proceed through the appropriations process for
writing a bill that actually is going to come to the floor next week
possibly. This will be the last appropriations bill considered by the
Senate this year. So we want to be sure we have our facts and arguments available to describe the reasons why we think funding of
additional medical research is so important to the future of our
country and mankind. Thank you for the contribution you have
made to that effort.
The hearing is recessed.
[Whereupon, at 10:31 a.m., Wednesday, October 19, the hearing
was concluded, and the subcommittee was recessed, to reconvene
subject to the call of the Chair.]

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